Health In Focus
  • Diabetic retinopathy is a complication of long standing diabetes mellitus, that involves the blood vessels in the eye.
  • With progression, complications such as retinal detachment and even blindness can occur.
  • Novel drug targets a protein that contributes to retinopathy, and could be a potential treatment.

Targeting RUNX1 protein, which is found in the abnormal vessels in the retina could reduce the severity of blood vessel involvement significantly, according to a preclinical study conducted at Massachusetts Eye and Ear by a team of scientists from Harvard Medical School.

Reason for the Study

  • The research team felt that current treatments for advanced eye disease in diabetes, though effective, can have complications.
  • They hope to make their drug safe and simple so that it can be self-administered by the patient, thus avoiding injections and other invasive procedures involved in current forms of treatment.
  • In addition, other diseases showing abnormal blood vessel growth in the eye such as wet age-related macular degeneration, cancer and retinopathy of prematurity can also be treated by their drug.

Methods and Findings of the Study

The current study is still in the nascent preclinical stage.
  • The study team found the occurrence of RUNX1 protein, which has been found to promote angiogenesis (new blood vessel formation) in the blood vessels affected by diabetic retinopathy, but absent in the normal blood vessels.
  • Inhibition of RUNX1 protein by a novel small-molecule drug showed a 50 percent reduction in the severity of retinal blood vessel involvement in preclinical models.

What is Diabetic Retinopathy?

In long standing diabetes, the blood vessels of the retina in the eye become affected. This is referred to as diabetic retinopathy. With progression of the disease, there is an abnormal increase in the number of blood vessels in the retina, referred to as proliferative diabetic retinopathy (PDR).
Retinal Microvascular Disease Could Be Prevented By New Drug

PDR can cause serious complications such as clouding of vision, increase in the intraocular pressure (glaucoma), retinal detachment and ultimately blindness. In fact diabetic retinopathy is a leading cause of blindness in the elderly.

How The New Drug Hopes To Score Over Current Treatments for PDR
  • Current treatments for proliferative diabetic retinopathy (PDR) involve laser treatments and injections that target a growth factor, the vascular endothelial growth factor (VEGF). In many cases, the eye injections have to be repeated as often as once a month.
  • These treatments are effective but complications such as retinal detachment and bleeding have been reported.
  • According to the authors of this research, the drug used to inhibit RUX-1 protein is a small molecule that could cross biological barriers independently, overcoming the need for painful and frequent injections.
  • In fact the treatment could be administered by the patient himself.
In the words of co-corresponding authorJoseph Arboleda-Velasquez, assistant professor of ophthalmology at Harvard Medical School and assistant scientist at Schepens Eye Research Institute of Mass. Eye and Ear, "Our study opens the door for new modalities of treatment based on small molecules that could cross biological barriers on their own. Such a treatment could be self-administered by patients and eliminate the need for intravitreal injections,"

Future Research Plans
  • The research team hopes to determine whether RUX-1 inhibition can target the disease process much earlier even before the condition becomes clinically apparent.
  • Future research plans include determining whether the drug can be delivered as topical eye drops rather than injections making the treatment safe, simple and effective.
  • Further, they wish to explore the relationship between RUX-1 and VEGF, since both appear to play a role in abnormal new blood vessel growth.


If the research work does manage to develop a drug that can be topically administered to treat and probably even prevent PDR, a dreaded complication of long standing diabetes, it would indeed be a path breaking achievement in the management of retinopathy.

In the words of co-corresponding authorLeo Kim, assistant professor of ophthalmology at Harvard Medical School and a retina surgeon at Mass. Eye and Ear, "We're hopeful that we may have an opportunity to change the treatment paradigm for these conditions. Instead of treating patients after these abnormal blood vessels form in the eye, we may be able to give patients eye drops or systemic medications that prevent their development in the first place."

References :
  1. Researchers identify new target for abnormal blood vessel growth in the eyes - (
  2. Diabetic Retinopathy - (

Source: Medindia

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